Biotech building facility to make genome-edited, off-the-shelf CAR-T therapies

Precision BioSciences, one of the biotechs working with so-called off-the-shelf chimeric antigen receptor T-cell (CAR-T) therapies, last year bagged $110 million in a series B round. Some of that cash has gone toward building out a manufacturing facility at its Durham, North Carolina, headquarters it says will be up and running by the end of the year. 

The facility will initially be used for its clinical work in oncology but has the potential to be a commercial launch facility with the capacity to generate up to 10,000 doses of CAR-T cell therapies and 4,000 doses of gene therapies per year, the company says.

"As part of our mission to overcome cancer and provide valuable new treatment options for patients, we are rapidly advancing a pipeline of next-generation, off-the-shelf CAR-T product candidates and we anticipate, once optimized, this platform will be able to support two new clinical programs per year," Precision BioSciences founder and CEO Matt Kane said in a statement. 

"Given the potential output of our platform, we’ve known from the beginning that it was critical for us to address the need for scalable manufacturing of cell-therapy products in order to be able to effectively deliver them to patients," he said.

A company spokesperson said the facility is part of a $26 million expansion Precision BioSciences has undertaken in North Carolina. The facility has 10 full-time employees with an anticipated capacity of 24, she said.

The facility is currently going through commissioning, qualification and validation ahead of its fourth-quarter launch. Precision says the multiproduct facility is designed to produce three different drug substances: allogeneic CAR-T cells, messenger RNA—including formulations development—and adeno-associated viral vectors.

So far, CAR-T products on the market are engineered and manufactured on a one-on-one basis, unique to each individual patient. That’s extremely expensive and time-consuming. The ability to have a single product available for appropriate patients would be a significant advance in the CAR-T market.

Precision’s CAR-T product candidates use T cells from qualified donors and then are manufactured in large batches and cryopreserved in order to be shipped and stored. They can then be used off the shelf for different treatments.

While there are other companies working in this area, Precision is built around Arcus, a genome editing platform that uses a synthetic mimic of the homing endonucleases found in nature to make insertions, deletions or other edits to DNA. The biotech believes its approach is more specific than competing gene-editing technologies as it reduces the risk of off-target activity. 

Precision’s facility will not be the only one in North Carolina dedicated to allogeneic CAR-T cells. In March, French biotech Cellectis announced it was working on an 82,000-square-foot commercial-scale manufacturing facility in Raleigh, North Carolina, to handle both clinical supply and commercial production of the allogeneic CAR-T treatments on which it is working. It is also building a 14,000-square-foot facility in Paris and says it will invest about $100 million in the two.